Research to optimise liver surgery for patients with neuromuscular diseases.

Abstract

Neurological and muscular disorders are uncommon conditions, and our understanding of their underlying causes and disease mechanisms has gradually improved over time. However, many general physicians often have limited knowledge about these disorders. When it comes to performing surgical treatments for liver diseases in patients with specific neuromuscular disorders, it can be challenging to apply the same treatment approaches as those used for patients without these disorders and to determine the most appropriate treatment methods.

The main goal of this research is to develop effective treatment strategies for pediatric patients who have neuromuscular disorders along with concurrent liver diseases that require surgical intervention.

For instance, in the case of patients with Myotubular Myopathy (MTM), their liver tissue is fragile, and removing a part of the liver (hepatectomy) may increase the risk of bleeding due to undetected peliosis hepatis in the remaining liver. In Japan, living-donor liver transplantation (LDLT) has been successfully performed for four MTM patients who experienced liver hemorrhaging caused by peliosis hepatis. Fortunately, these patients have had positive outcomes after the surgery. By reviewing these cases, our aim is to investigate both short-term and long-term complications and gain a better understanding of how LDLT influences the development and overall prognosis of these pediatric patients. Additionally, since these diseases are extremely rare, we plan to conduct basic research to gather as much knowledge as possible.

Furthermore, we aim to expand our research to include other patients with neuromuscular disorders to identify any specific considerations when treating liver diseases in this patient population.

Through collaboration with experts in neurological and muscular disorders, we can offer appropriate surgical treatments for liver diseases in patients with neuromuscular disorders.

Perspectives

Establishing treatment strategies for liver disease in paediatric patients with neuromuscular disease.

Investigating the impact of treatment strategies for liver disease on the prognosis of the patients.

Comments from principal researcher

Organ Transplantation Center, National Center for Child Health and Development, Seiichi Shimizu

Several years ago, we performed LDLT for the MTM patients with massive bleeding due to peliosis hepatis. As surgical specialists, neurological and muscular disorders are not our usual area of expertise. However, by collaborating with specialists in the field of neurology, we have conducted in-depth analysis of these cases and engaged in basic research. Through this approach, we aim to establish treatment strategies and provide medical care across different specialties, with the concept of "healing patients with liver diseases.

Shared Researchers

Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry (NCNP), Tokyo, Japan.　Kaoru Yamamoto.